FDA Approves Novartis' Fabhalta for Rare Kidney Disease

Novartis' innovative therapy, Fabhalta, has secured another significant endorsement from the FDA, this time for addressing an extremely rare kidney condition known as C3 glomerulopathy (C3G). This condition progressively deteriorates the small filters within the kidneys, potentially resulting in kidney failure and the necessity for lifelong dialysis. Fabhalta, now recognized as the first approved treatment specifically targeting C3G, is administered in capsule form twice daily.

This recent approval marks Fabhalta's third regulatory success. In late 2023, the medication was approved for treating paroxysmal nocturnal hemoglobinuria, a rare blood disorder. More recently, it received accelerated approval for use in patients with IgA nephropathy, another kidney-related disease.

The FDA's endorsement for treating C3G was based on findings from a Phase 3 clinical trial. This study demonstrated that, when combined with supportive care, Fabhalta led to a 35% reduction in proteinuria after six months compared to a placebo, successfully achieving the study's primary objective. Novartis further reported in October that this reduction in proteinuria was maintained over a period of one year.

C3G involves the abnormal activation of a component of the immune system known as the complement system, resulting in the accumulation of proteins in the kidney's small filters. Fabhalta functions by inhibiting a specific protein involved in this system, thereby mitigating the disease's progression.

Initially, the FDA planned an advisory committee meeting to review Fabhalta's application for C3G, but this meeting was unexpectedly canceled in February. Victor Bulto, Novartis’ US President, explained that the FDA initially required six months of data, later requesting 12 months' worth, which was duly submitted. Subsequently, the FDA communicated that they no longer considered an advisory meeting necessary, without providing further specifics.

When questioned about whether this cancellation was linked to the new Trump administration, Bulto stated his lack of insight on the matter, indicating that the company only received standard FDA communication regarding the decision.

Novartis is actively expanding its portfolio of treatments for kidney diseases, starting with Fabhalta. The company is also anticipating an FDA decision on another drug, atrasentan, targeting IgA nephropathy, expected during the first half of the year. Additionally, Novartis is conducting Phase III studies on zigakibart, an anti-APRIL antibody, for the same condition. Both atrasentan and zigakibart were acquired through Novartis' strategic purchase of Chinook Therapeutics in 2023, a deal valued at $3.2 billion.

Overall, Novartis' efforts represent a significant advancement in addressing rare kidney diseases, underscoring the company's commitment to developing and providing innovative treatments for conditions with limited therapeutic options. Fabhalta's latest approval not only broadens the treatment landscape for C3G but also emphasizes the potential of targeted therapies in managing complex kidney disorders.